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Targeting Oligodendroglial Dysfunction in Angelman Syndrome

$50,500 2023 PD2023-001

Targeting Oligodendroglial Dysfunction in Angelman Syndrome

Stem cell technology could prove invaluable in discovering effective treatments for developmental brain disorders, including Angelman syndrome (AS). In the brain, information of learning and memory is processed and stored in the form of electrical signals transmitted through millions of circuits made of neurons. The supporting glial cells, such as oligodendrocytes, are active partners in such information processing by providing structural support to neurons to speed up the flow of electric signals. In AS, this function of oligodendrocyte falls is impacted in children and young adults, and may slow the brain development and increase seizures. This project aims to understand and translate how the unique biology and genetics of AS make a difference in the oligodendrocyte function. Specifically, a few selected compounds identified from a stem cell-based drug screen will be tested for their efficacy by using a mouse model and the induced pluripotent stem cells (iPS cells) donated from AS patients and health individuals.

Principle Investigator

Xin Yang, PhD and Yu-Wen Alvin Huang, PhD